Treatment with Ataluren for Duchene Muscular Dystrophy.
Journal: Pediatric Neurology Briefs
Published:
Abstract
Investigators from Europe and the USA, representing the STRIDE Registry and Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), examined the effectiveness of ataluren and standard of care in the Registry versus stand of care alone in the CINRG DNHS.
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